Peptide Receptor Radionuclide Therapy (PRRT) with 177 Ludotatate in Metastatic Phaeochromocytomas and Paragangliomas – a single centre retrospective analysis of experience at an ENETS centre of excellence

Introduction: 177Lu-DOTATATE peptide receptor radionuclide therapy (PRRT) represents a possible therapeutic option for patients with metastatic inoperable phaeochromocytomas (PCC) and paragangliomas (PGL) who demonstrate adequate somatostatin analogue binding on molecular imaging. We describe treatment outcomes in our cohort of patients stratified according to germline pathogenic variants (PV) in Succinate Dehydrogenase (SDHx) subunit-encoding genes. // Methods: In this retrospective analysis, we evaluated 20 patients with metastatic PCC/PGL who underwent two or more cycles of 177Lu-DOTATATE therapy. Clinical, radiological, and biochemical responses were assessed 8-12 weeks after the final PRRT cycle. We describe overall treatment efficacy at follow-up after stratifying according to the presence of germline SDHx PV. Radiological progression was quantified based on the sum of the longest diameter (SLD) of the target lesion. Progression-free survival (PFS) and overall survival (OS) were estimated using Kaplan-Meier survival analysis. We also aimed to investigate the impact of PRRT on health-related quality of life (HRQoL), as assessed using the EORTC QLQ-GINET21 questionnaire. // Results: After a median follow-up of 29 months, we confirmed stable disease in 12 patients (60%), a partial response in one (5%), and progressive disease in 7 patients (35%). The absolute mean difference in SLD was +5±12 mm for bone lesions, -4±6 mm for peritoneal, +8±14mm for liver lesions and -1± 5 mm for lymph nodes (paired t-test p-value 0.273, 0.741, 0.208 and 0.826, respectively). Thirteen patients (65%) had received two or more previous lines of treatment. The overall median PFS for the entire cohort, PGL patients, SDHx positive and negative groups, was 24 months (95% CI, 9.9-38.1), 18 months (95% CI, 8.4-27.6), 24 months (95% CI, 11.9-36.0) and 18 months (95% CI, 0-48) respectively. No grade 3/4 cytopenia or nephrotoxicity was observed. Overall, HRQoL improved after PRRT, as evidenced by the progressive decline in overall symptom scores in the QLQ-GINET21. // Conclusion: 177Lu-PRRT appears to be an effective therapy with a good safety profile for patients with metastatic PPGL. It also appears to improve HRQoL in patients with metastatic PPGL. Further studies are needed to explore the most effective treatment modalities in this group of patients and their sequencing

The association of hyperthyroidism and immune thrombocytopenia: Are we still missing something?

Though the American society of hematology Guidelines and British guidelines do not recommend screening of thyroid diseases in cases of immune thrombocytopenia (ITP), more than 160 cases of hyperthyroidism associated with ITP have been reported. Numerous case reports would suggest that patients with ITP and concurrent hyperthyroidism would respond to control of thyroid disease rather than the standard ITP treatment. Although this issue is still debatable, we report a case of a young female with a previous diagnosis of hyperthyroidism which was not well controlled, had presented with severe thrombocytopenia. Initially, all work-up had been done to find out the cause of thrombocytopenia. After all normal reports except deranged thyroid function tests, the patient was labeled as ITP and started on steroids. Even after a few months of steroid treatment, platelet counts had not improved. However, after starting antithyroid drugs, platelet counts had become normalized

Guillain-Barré syndrome presenting with COVID-19 infection

A construction worker in his 30s presented three times in 4 days with progressive upper and then lower limb weakness. On the first two occasions he had no systemic symptoms, but on the third presentation he had fever and cough, starting from day 4 of weakness. Examination identified weakness in all four limbs and areflexia, suggesting a peripheral neuromuscular disorder. Investigations were consistent with Guillain-Barré syndrome and additional COVID-19 (SARS-CoV-2) infection. The patient improved after immunoglobulin treatment. At least four cases of Guillain-Barré syndrome have been reported in the literature with concurrent COVID-19 illness in whom respiratory signs appeared a few days after the onset of neurological signs. With the incubation period for COVID-19 respiratory symptoms believed to be up to 14 days, it is possible that neurological symptoms could develop before respiratory and other symptoms. During the current pandemic, presence of concurrent COVID-19 infection needs to be considered in patients presenting with Guillain-Barré syndrome.</jats:p